PM360

APRIL 22, 2024

In the rapidly evolving landscape of healthcare, where breakthrough therapies and treatments emerge at an unprecedented pace, conventional boundaries are rapidly shifting. Understanding the “why” before the “how” as Med Affairs charts the course for pre- and post-launch strategies. Medical Affairs is at the forefront of this paradigm shift.

Medical 52

Medical Healthcare Healthcare Education 52

Pharmaceutical Technology

JUNE 29, 2022

This year has already been eventful when it comes to the development of therapies for rare diseases. Additionally, pricing and access for rare disease therapies continue to be scrutinized closely. Additionally, pricing and access for rare disease therapies continue to be scrutinized closely.

Food and Drug Administration 98

Food and Drug Administration FDA Marketing Pharmaceutical 98

pharmaphorum

NOVEMBER 16, 2022

The advancements made in drug development over the last decade have seen the arrival of a number of treatments that offer one-time cures and more effective therapies for conditions in smaller patient populations. However, alongside this progress, Ben Hargreaves finds that there is also a conundrum over how to price these therapies.

Medicine 120

Medicine Marketing Healthcare Healthcare 120

Pharmaceutical Technology

JUNE 21, 2023

CSL announced the first patient with haemophilia B has been treated with its recently approved gene therapy Hemgenix (etranacogene dezaparvovec). Hemgenix is the first and only gene therapy approved for haemophilia B. per dose, it is the most expensive single-use gene therapy in the US.

Patients 98

Patients Food and Drug Administration Engineering Sales 98

European Pharmaceutical Review

MAY 11, 2023

Novartis shared long-term data for its SMA gene therapy in March 2023, which indicated significant long-term motor-milestone achievements in spinal muscular atrophy (SMA). Without this protein, the motor neuron cells shrink and eventually die. This causes debilitating and potentially fatal muscle weakness. Why is it significant?

Food and Drug Administration 72

Food and Drug Administration Government Healthcare Healthcare 72

pharmaphorum

AUGUST 26, 2022

BioMarin Pharma has its landmark first approval for Roctavian – the first for a haemophilia gene therapy – and will now have to see if that can convert that into a viable business in Europe. BioMarin has said Roctavian will be available at a ‘net’ price of around €1.5 million spread over five years.

FDA 108

FDA Marketing Competition Patients 108

PM360

APRIL 7, 2023

The shift toward longer, more adequate timeframes to establish proof of concept can be as long as three to four years, 1 given the complex requirements for developing and launching specialty drugs and cell and gene therapies (CGTs) for individuals with complex conditions or rare or orphan diseases.

Manufacturing 52

Manufacturing Marketing Patients Safety 52

European Pharmaceutical Review

DECEMBER 27, 2023

Chimeric antigen receptor (CAR) T cells are the first example of a “living drug”. 1 Following in vitro production from T cells collected from a patient’s leukapheresis procedure, the CAR T cells are infused back into the patient’s blood, where they proliferate and expand.

Manufacturing 85

Manufacturing Food and Drug Administration Patients Pharma 85